EZN-4482

EZN-4482 is a locked nucleic acid (LNA)-based antisense oligonucleotide (ASO) designed to target and down-regulate GLI2 mRNA, thereby inhibiting the Hedgehog signaling pathway. Developed by Enzon Pharmaceuticals in collaboration with Santaris Pharma, EZN-4482 specifically blocks the expression of GLI2, a key transcription factor and downstream effector of the Hedgehog pathway implicated in tumor initiation, growth, and resistance. In preclinical studies, systemic administration of EZN-4482 demonstrated potent down-modulation of GLI2, GLI1, and PTCH1 in both tumor and host stromal cells, resulting in tumor growth inhibition in prostate cancer xenografts and prolonged survival in lung carcinoma liver metastasis models.